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NCT03364153CompletedPhase 2

Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease

Source: ClinicalTrials.gov · Last updated 15 Apr 2026

Brief summary (registry, in English)

The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).

Status (registry)
Completed
Phase (registry)
Phase 2
Study type
Interventional
Enrollment
121 (actual)
First submitted
1 Dec 2017
Start date
12 Jan 2018
Primary completion date
31 Mar 2025
Lead sponsor
Astellas Pharma Global Development, Inc.
NCT number
NCT03364153
EudraCT number
2017-004783-35

What you commit to

  • Study type: Interventional · Phase 2
  • Participants enrolled: 121
  • From 12 Jan 2018 to the primary completion date, 31 Mar 2025
  • 41 sites in 8 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
  • Participation in an interventional study of a vitamin A derivative \</= 3 months prior to screening
  • Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
  • Any major surgical procedure within one month of trial entry or anticipated during the trial
  • Any treatment with an investigational agent in the past 60 days for any condition

Other illness

  • Macular atrophy secondary to any condition other than STGD1 in either eye
  • Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
  • Diabetes mellitus
  • Stroke within 12 months of trial entry
  • Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the avacincaptad pegol formulation

Lab values

  • Hemoglobin A1c (HbA1c) value of \>/=6.5%

Other

  • Women who are pregnant or nursing

Who the study is looking for

  • At least two pathogenic mutations of ATP-Binding Cassette (ABC)A4 gene confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
  • Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive

Age in the registry: 18 years to 60 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (41)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
University of Bonn
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BonnGermanyUnknown status
Augenklinik der LMU München
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MünchenGermanyUnknown status
University of Tuebingen
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TübingenGermanyUnknown status
Institut de la Macula
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BarcelonaSpainUnknown status
Creteil University Eye Clinic University Paris EST
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CréteilFranceUnknown status
Hôpital de la Croix Rousse
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LyonFranceUnknown status
Centre ophtalmologique des Quinzes Vingts
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ParisFranceUnknown status
Princess Alexandra Eye Pavillion
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EdinburghUnited KingdomUnknown status
Moorfields Eye Hospital
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LondonUnited KingdomUnknown status
Budapest Retina Institute
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BudapestHungaryUnknown status
Semmelweis Egyetem
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BudapestHungaryUnknown status
University of Debrecen DE KK Szemészeti Klinika
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DebrecenHungaryUnknown status
Ganglion Medical Center
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PécsHungaryUnknown status
Szegedi Tudományegyetem Szent-Györgyi Albert Klinikai Központ, Szemészeti Klinika
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SzegedHungaryUnknown status
Rambam Health Care Campus
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HaifaIsraelUnknown status
Hadassah University Hospital
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JerusalemIsraelUnknown status
Rabin Medical Center-Beilinson Campus
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Petah TikvaIsraelUnknown status
Kaplan Medical Center
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RehovotIsraelUnknown status
Tel Aviv Sourasky Medical Center (Ichilov Hospital)
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Tel AvivIsraelUnknown status
AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
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BolognaItalyUnknown status
Azienda Ospedaliera Universitaria Careggi
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FlorenceItalyUnknown status
Ospedale San Raffaele
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MilanItalyUnknown status
University of Campania Luigi Vanvitelli Eye Clinic
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NaplesItalyUnknown status
Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche
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RomeItalyUnknown status
University of Michigan Kellogg Eye Center
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Ann ArborUnited StatesUnknown status
Austin Retina Associates
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AustinUnited StatesUnknown status
Wilmer Eye Institute Johns Hopkins
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BaltimoreUnited StatesUnknown status
Retina Center of NJ, LLC.
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BloomfieldUnited StatesUnknown status
Ophthalmic Consultants of Boston
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BostonUnited StatesUnknown status
Retina Foundation of the Southwest
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DallasUnited StatesUnknown status
VitreoRetinal Associates
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GainesvilleUnited StatesUnknown status
Jules Stein Eye Institute/ David Geffen School of Medicine
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Los AngelesUnited StatesUnknown status
The Retina Center
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MinneapolisUnited StatesUnknown status
Retina Specialty Institute
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PensacolaUnited StatesUnknown status
Wills Eye Hospital/Mid Atlantic Retina
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PhiladelphiaUnited StatesUnknown status
Retinal Research Institute
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PhoenixUnited StatesUnknown status
UPMC Eye Center
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PittsburghUnited StatesUnknown status
Casey Eye Institute, Oregon Health & Science University
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PortlandUnited StatesUnknown status
University of Utah John A. Moran Eye Center
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Salt Lake CityUnited StatesUnknown status
Palmetto Retina Center
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West ColumbiaUnited StatesUnknown status
Strategic Clinical Research Group
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Willow ParkUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.